Canada’s Gaucher Drug Bidding War

The standard of care for Canadians with Gaucher disease just changed. The transformation will likely reduce the drug costs per patient by tens of thousands of dollars per year and it is all due to the pan-Canadian Pharmaceutical Agency (pCPA).

For over 20 years, the gold standard for treating Gaucher disease type 1 was Sanofi Canada’s Cerezyme (imiglucerase), an enzyme replacement therapy (ERT) that is infused every couple of weeks into the person with Gaucher disease to replace the enzyme they are missing.

There are 2 other ERTs available in Canada for Gaucher disease—Shire’s Vpriv (veraglucerase alfa) and Pfizer’s Elelyso (taliglucerase alfa) but a large majority of Canadians with Gaucher disease were taking Cerezyme.

The 3 ERTs have slight differences but overall, the 3 have fairly similar safety and efficacy rates.

These ERTs require biweekly infusions involving a healthcare professional (either a nurse who comes to the person’s home or a nursing team at an infusion centre). And all 3 are orphan drugs for a rare disease. In other words, they are expensive.

Recently, the pCPA office sent notices to the makers of the Gaucher disease drugs to compete in what Christine White, president of Gaucher Canada called a ‘tender bid.” White said, “Shire, Sanofi-Genzyme, and Pfizer all presented to the pCPA with their best and lowest price. Months went by and then in June, pCPA informed the companies the breakdown of how patients with Gaucher disease will be treated.”

And the winner of the bidding war was Shire. Moving forward, patients with Gaucher disease in a public plan will first be given Shire’s Vpriv. And if that drug proved ineffective or unsafe for the patient, their second choice will be Pfizer’s Elelyso followed by Sanofi-Genzyme’s Cerezyme.

The presence of Elelyso on the list was surprising given that the drug did not get a favourable rating by CADTH in its review. Also, surprising was the fact that pCPA lumped the oral substrate replacement therapy, Cerdelga (eliglustat), with the ERTs and that drug is not even listed as an option by pCPA. White said, “so unless you have private insurance, that oral drug is not an option.”

When will the Switch to VPriv Occur?

White said she recently talked to a physician in British Columbia and in that province, patients taking Cerezyme are already being switched to Vpriv. “They are not being given a choice. They have to do it,” said White.

However, with all this secrecy and abruptness, White noted that no patients have yet to complain about the changes.

In other provinces, the switch may be delayed, especially in Ontario. “Years ago, when we lobbied for access to Cerezyme, it was written into the legislation that the Ontario budget would provide access to this drug,” stated White. But that may change. White added, “pCPA are working on changes to the legislation. So it might be another year before that gets straightened out.”

Is the pCPA doing a good job?

So far, the answer is yes. White thinks that they are significant financial savings to the health care system as a result of the recent negotiations and to date, no patients have complained about being forced to switch medications.

Of concern to White is lack of input pCPA requested from the patient or medical community. Given that Gaucher disease is a rare condition, it is unlikely that members of the pCPA are very familiar with pros and cons of the various treatment options. White said, “I reached out to them once I became aware of the negotiations. I got a meeting with Imran Aliat the pCPA and I brought Drs. Dominick Amato and Mary Anne Patterson from the Gaucher Centre in Toronto.”

Whether or not that meeting had any impact is not clear but White noted, “the fact that we were not consulted, nor was any patient consulted during this process, was concerning.”

The lack of openness may not be confined to just these negotiations. A google search of pCPA shows very limited results. They are part of the Canada Premiers’ website and when you go to their webpage, the top section has news and information dated from 2016 and it is not until you scroll to the very bottom of the page, that you can find links to drugs currently being negotiated, drugs that have been negotiated, drug not be negotiated, and drugs being considered for negotiation. Additionally, those lists do not include any further information about the negotiations. In an era where the Canadian government is rallying to have more transparency in the way drug prices are established, the degree to which pCPA seems to be non-transparent is somewhat surprising.

As for the oral, non ERT product, Cerdelga, White informed us that cPCA plans to conduct a 30 minute call with Sanofi-Genzyme to discuss that drug as a treatment option. “I don’t know what can be resolved in a half hour call,” stated White but she is curious to see if that call is enough to prompt cPCA to look at the Cerdelga application again. The oral drug is likely more expensive but does not require regular nurse-supervised infusions that are needed for ERTs.